On Nov. 21, 2016, the American Society of Gene & Cell Therapy (ASGCT) released Therapeutic Gene Editing: an ASGCT White Paper, intended to provide policy-makers, patient advocates, and the interested public with the necessary background information in anticipation of an upcoming consensus report on human gene editing from the National Academy of Sciences and National Academy of Medicine (NAS), expected to be released in early 2017.
Researchers, physicians, and patients believe gene editing can greatly improve the outlook for currently incurable genetic diseases, such as muscular dystrophy, sickle cell disease, cystic fibrosis, and hemophilia, among many others. Genetic diseases are caused by mutations in genes and currently impact more than 30 million Americans, 2/3 of whom are children. Gene editing seeks to correct or change genes only in cells in which the alteration is expected to have a therapeutic benefit to patients.
The drug treatments currently approved for genetic diseases only manage symptoms, they do not address the underlying cause of the disease. "The successful development of gene editing applications could shift a lifetime of symptom management towards tomorrow's ideal of a one-time curative treatment for patients," said Dr. Cynthia Dunbar, ASGCT's President, noting that the process is monitored closely by global regulators including the FDA and their European counterparts.
A cohort of ASGCT member scientists traveled to Washington on Nov. 18 to present the Society's educational materials to relevant congressional committees and administrative agencies, including the U.S. Senate Health, Education, Labor, and Pension (HELP) Committee and the White House Office of Science and Technology. The goal of these meetings was to educate policy-makers on the fundamentals of gene editing.
The NAS consensus report, which is slated to be released in early 2017, is expected to address ethical, legal, and social implications of gene editing. ASGCT believes its November white paper will be a useful tool to assist advocacy groups, lawmakers, and the news media to better interpret the 2017 NAS consensus report.
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